AAV6-mediated Systemic shRNA Delivery Reverses Disease in a Mouse Model of Facioscapulohumeral Muscular Dystrophy

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Medicine, Genetics, Natural Sciences +8

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

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Effective expression of small interfering RNA in human cells

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Effective Expression, Small Rna, Gene Expression +3

Short GCG expansions in the PABP2 gene cause oculopharyngeal muscular dystrophy

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Systems Biology, Genetic Disorder, Short Gcg Expansions +7